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The FDA’s August approval of Takeda’s Orzeyful for treating the full set of narcolepsy symptoms initiated a new era in the sleep market.
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If we guard our secrets, patients suffer. Only through a commitment to transparency and data sharing—and artificial intelligence—can we make cell and gene therapies safer and more effective.
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
Companies that manufacture MDMA and psilocybin for patients in other countries are looking to use their experience to access the potential U.S. market.
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The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.
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The discontinuation of the uveitis study adds to Regeneron’s series of clinical setbacks, including the Phase 3 failures of the Dupixent follow-up itepekimab and an investigational combo regimen for melanoma.
With a Phase 3 trial showing a reduction of serious and severe hypoglycemic episodes in patients who have undergone weight loss surgery, Amylyx Pharmaceuticals plans to file for approval this year. If granted market entry, avexitide could hit peak sales of $1.7 billion worldwide, analysts estimate.
EyePoint’s quest to reduce treatment burden for patients with age-related macular degeneration has hit a bump in the road, with a clinical miss driving the biotech’s stock down by 70%.
Ivonescimab notched its third approval in China last week, but the label update has tempered expectations for the Summit Therapeutics/Akeso drug, showing that progression-free survival declines over time.
AstraZeneca will push through with its other Phase 3 studies for volrustomig, including in cervical cancer and mesothelioma.
Otsuka Pharmaceuticals’ Voyxact is not the only IgA nephropathy drug that can slow the decline of kidney function. Vera Therapeutics’ Trutakna, which the FDA granted accelerated approval last month, showed “impressive” stabilization, analysts say.
Johnson & Johnson secured rights to prizlon-cel in 2023 for $245 million upfront. The asset in June last year showed a 100% response rate as a second-line option in a Phase 1b study for 10 patients with relapsed or refractory large B cell lymphoma.
Despite ranking among the world’s top scientific producers, Italy has struggled to turn research into startups. Now, stronger tech transfer offices, private capital and returning scientists are pushing the country toward a more mature innovation economy.
The FDA approves Zenbexus just as copycat competitors depress sales of Bristol Myers Squibb’s multiple myeloma drugs Pomalyst and Revlimid, which crashed 71% and 49% year-on-year, respectively, in the second quarter.
Sponsors of the pivotal study behind Amgen’s rare disease drug Tavneos saw unblinded findings for the drug and re-adjudicated data from nine patients to make the drug seem effective, according to European regulators.